Parents are using nearly 400 different treatments for their children with autism. They can't all be wrong. They can't all be right.
Welcome to the shaky ground on which parents find themselves when they learn that their child may -- or may not -- have autism.
The pace of scientific research is frustratingly slow. Many treatments that seem to make sense -- and that other parents swear by -- haven't been proven effective or safe, ineffective or harmful. Compounding this confusion, any number of charlatans stand ready to offer spurious cures.
"The information was so overwhelming and scary," remembers Debbie Page, whose son Gabe was diagnosed with autism in 2005. "It was a scary time of 'What is right?' 'What is real?' 'What do I need to focus on right now?'"
Paul A. Law, MD, MPH, and Kiely Law, MD, MPH, researchers at the Kennedy Krieger Institute (and parents of Isaac, a child with autism), last year launched the Interactive Autism Network (IAN). It's already enrolled the families of nearly 8,000 children with autism, offering targeted enrollment in research studies, rapid feedback on what is learned, and networking opportunities.
"Quite a number of these children are on more than 30 or 40 treatments at any given time, not including everything else they may have tried and stopped using," Paul Law tells WebMD. "One child is on 56 treatments at one time."
One problem is that as claims proliferate, it's difficult for parents to separate the wheat from the chaff, says autism researcher Susan Hyman, MD, of the Strong Center for Developmental Disabilities at the University of Rochester, N.Y.
"It's back to the future in autism: Everything that anybody has ever tried, from guided imagery to vitamins, is still out there," Hyman tells WebMD. "On the Internet, there is a tremendous explosion of information. But I don't know there is any more capacity to discern medically reviewed data from other data. And physicians are terrible at marketing. Evidence is just not as effective as advertising."
At the heart of the issue is the fact that what most people call "autism" is actually a spectrum of disorders that may or may not turn out to have different causes. That's why experts prefer the term autism spectrum disorder or ASD.
Normally, this includes the specific diagnoses of autistic disorder, Asperger's syndrome, and pervasive developmental disorder-not otherwise specified or PDD-NOS. One thing that complicates autism research is that different autism spectrum disorders may turn out to have different causes, may respond better to different treatments, and, perhaps one day, will have different cures. Today, however, ASD has no known cause, no one-size-fits-all treatment, and no cure.
Earlier Autism Treatment Is Better Autism Treatment
Perhaps the biggest breakthrough in autism treatment to date is the recognition that pediatricians can identify most (but not all) 24-month-old and even 12-month-old children with autism.
Why is this such a big deal? Just about everybody agrees that whatever it is that goes wrong in autism goes wrong in the brain. And while a child's brain continues to develop through the teen years, the most intensive period of change is the early years of life.
And now researchers are finding effective treatments for young children. One is Rebecca Landa, PhD, director of the Center for Autism and Related Disorders and the REACH research program at Baltimore's Kennedy Krieger Institute.
Landa's current project is her Early Achievements program, which extends individualized, behavior-oriented autism treatment to 2-year-old children. At this age, most children with autism get a weekly or monthly visit from a therapist who trains parents to do behavioral interventions in the child's natural environment.
They get far more in Landa's classes, in which a small number of children get both one-on-one and group experiences. This is a challenge for any child this young, but a particular challenge for kids with autism, who face a range of problems with communication and social skills. They may have trouble learning to talk, imitating others, sharing emotions, and paying attention. They may show interest in a very few things. They may engage in repetitive, self-stimulating behaviors (which parents and autism professionals often call "stimming.")
"They are still babies. It is usually the first time they have been away from their parents -- this is very hard for kids with autism," Landa tells WebMD. "We start with, not a blank slate, but with very raw material. The challenge for us is to choose the right toys and deliver them in the right activities to draw these kids attention and keep it for more than 30 seconds. And then we must be patient as these kids resist being with us and with other kids. We are constantly reassuring them until they get to the point where they are able to initiate interactions with other kids."
Behavior therapy targeted to the individual child's needs is at the forefront of treatments researchers are trying today with ASD kids. Of all the treatments that parents try for their child, behavior therapy is the only one scientifically shown to help children with autism.
"Nobody responsible in the field say this cures autism, but many of these children can be improved substantially, dramatically, and some -- a very small percentage -- improve to the point you could not differentiate them from typical individuals," says Laura Schreibman, PhD, director of the autism research program and distinguished professor of psychology at the University of California, San Diego.
In Landa's program, it focuses nearly as much on parent and family training as it does on the child with autism.
"When you first get a diagnosis of autism, you are not ready for that. Your world is shaken. And suddenly your child is not who you thought they were. 'How do I play with my child?' 'How do I understand who my child is?' 'What do I do about it?'" Landa says. "We teach them the beauty within their child."
Every week the parents have to tell the class something wonderful about their child. At first, most parents cant think of anything.
"A week or so later, they can't wait to come in and tell us what wonderful thing their child did last. This lets parents focus on what is good, instead of something panicky," Landa says. "We teach them how to interact with their child in helpful, fun ways. We take care of the whole family and it is very powerful."
Debbie Page and her son Gabe enrolled in Landa's experimental program. Gabe had been diagnosed with "mild" autism -- but when Page heard what Landa expected the kids to learn, she was more than doubtful.
"I remember her saying the kids would transition themselves from one activity to another by checking their picture schedule and singing a little song," she says. "All the parents were nodding and I nodded, too, but inside I thought, 'There is no way he will do this.' My son screamed any time a demand was placed on him -- he didn't even respond to his name. I thought we'd be the first ones to get kicked out of the study."
Not two weeks later, Page got a call from Gabe's teacher saying that her son had checked his schedule all by himself.
"I knew then I would never say 'No way' about Gabe again. He has continued to amaze us," she says. "At first he did not know how to play with toys -- he didn't understand what play was. Six months later, he was engaging in play with other children. My father describes it as a light switch being turned on. ... I had never heard Gabe sing. The best he could do was to make a hand motion when I sang The Wheels on the Bus. But after six months, he was a songbird. It was really amazing."
Landa warns that not every child makes this kind of progress. However, she says that more than 60% of the kids in the program gain six months of language skills during the six-month program. That's not bad, given that the kids did not yet have 12-month language skills at an average age of 27 months. And Landa says a "large number" of the students gained 12 months of language skills during the program.
Do these gains endure? Landa says there's strong evidence they do, although the program only began in 2005. Gabe, now 5 years old, was fortunate enough to graduate into Baltimore County school programs with Kennedy-Krieger-trained teachers. This year, his teachers placed him in a regular pre-kindergarten program in a class of 20 children.
"By this kind of early intervention at age 2 -- and now we have a study with 1-year-olds -- when you get them really young and teach them how to learn, they are different kids," Landa says. "What would happen if you waited until they were 3? I wonder how much more capable we could make them by starting even earlier."
Drug Treatments for Autism
Unfortunately, many children with autism aren't able to enter any kind of behavioral or educational treatment. Some of these kids respond with violence or tantrums to any attempt to interrupt their obsessive "stimming" behavior. For some, this self-stimulation takes the form of self-injury. Other children with autism are hyperactive.
Might psychiatric drugs calm these symptoms enough to allow such children to enter behavioral and educational programs? Yes, says Yale's Lawrence David Scahill, MSN, PhD, a leader in pediatric psychopharmacology research.
Scahill was part of an NIH-funded group that showed that the anti-psychotic drug Risperdal could calm extreme behavior in autism spectrum disorder kids.
"Some 20% to 30% of school age kids with ASD, down to age 5 years, have problems with aggression, tantrums, or self-injury -- we thought that would be a good target for Risperdal," Scahill says. "We enrolled children with autism and at least moderate levels of tantrums -- not the kid who flops a little bit, but kids with outbursts you can measure on the Richter scale. They are not going to learn to toilet themselves or to play with toys. We thought if we could give these kids a medication, maybe they would be more malleable to other interventions."
The result was surprising -- kids who got the drug had a 58% improvement in this behavior, compared with 12% getting placebo.
"It was a big difference, the kind of difference we don't see in child psychiatry very often," Scahill says. "We credit it first to the drug, but also to the fact we only enrolled children with moderate or higher levels of this behavior. "
As a result of this study, the FDA approved Risperdal for treatment of irritability in kids with autistic disorder with symptoms of aggressive behavior, deliberate self-injury, or temper tantrums. Now Scahill and colleagues are trying to find out how soon kids can be tapered off the medication -- and whether parent training improves outcomes for kids receiving the drug.
Getting off Risperdal will be important, Scahill says, because a major side effect of the treatment is unhealthy weight gain.
A subsequent study looked at whether hyperactive kids with autism respond to Ritalin as well as ADHD kids without autism. The important finding: While 75% to 80% of ADHD kids without autism do better on Ritalin, this happens in only about 50% of hyperactive kids with autism. And the improvement in kids with autism wasn't as large as the improvement in kids without autism.
A more recent study is looking at whether the antidepressant Celexa, which helps control symptoms of obsessive-compulsive disorder, can reduce repetitive behaviors in children with ASD. Results of that study are expected soon.
Scahill notes that all of these studies have looked for ASD symptoms that match symptoms for which psychiatric treatments exist. Now, however, researchers are cautiously exploring a larger goal -- treating autism itself.
That's a problem, because nobody knows exactly what causes autism. But there are some exciting leads, says Susan Swedo, MD, chief of the pediatric and developmental neuropsychiatry branch of the National Institute of Mental Health.
One exciting avenue of research, Swedo says, is the glutamate system -- a chain of chemical messengers and receptors that represents one of the brains communication channels. This brain circuit is important in Lou Gehrig's disease, for which a glutamate-blocking drug called Rilutek is helpful.
Based on evidence that the glutamate system is overactive in childhood obsessive-compulsive disorder, Swedo and colleagues tried treating OCD kids With Rilutek.
"It was remarkably effective," Swedo tells WebMD.
If it worked in childhood OCD, perhaps it will help control repetitive behavior in children with autism, Swedo suggests. Scahill agrees this is possible.
"This is not pie in the sky. There is a lot of interest in the glutamate system. It is highly relevant to schizophrenia, and probably relevant to autism," Scahill says.
Yet another intriguing possible future treatment for autism is a brain molecule called oxytocin.
"Oxytocin is a naturally occurring hormone involved in labor and delivery that also plays a crucial role in attachment and early infant bonding," Swedo says. "It is kind of intriguing because we have this clue from baby mice genetically engineered to lack oxytocin -- they act like the mother mouse is a stranger. So here in autism you have kids who get into stranger anxiety. What if these kids had an oxytocin problem? It is an interesting clue."
A study of synthetic oxytocin infusions in adults suggested it might reduce repetitive behaviors; further research continues.
Both Swedo and Scahill warn that only step-by-step scientific research can show whether these new treatment ideas work. They point to what happened with secretin, a hormone once hailed as an autism cure.
Spurred by huge numbers of parents giving secretin to their ASD kids, researchers rushed to study the drugs effects.
"Secretin is right now the best-studied drug in autism," Scahill says. "There have been 12 or 13 placebo-controlled trials, but not one showed secretin to be better than placebo. Researchers spent vast amounts of time and money on it and we don't have a lot to show for it. That is an example of how it shouldn't go."
Chelation for Autism
Although most researchers do not think so, many parents are struck by similarities between some of the symptoms of mercury poisoning and autism. Some of these parents seek chelation therapy for their children, which uses a chemical that helps the body eliminate heavy metals.
Hyman notes that there is no evidence that removing heavy metals from the body undoes damage caused by heavy-metal poisoning. But many parents believe their children's ASD symptoms improved after the treatment.
Swedo and colleagues at the NIMH have designed a clinical trial to test this treatment, but the study is in limbo as the NIMH review board feels the known risks of the treatment outweigh the evidence that it might work. Meanwhile, Swedo says, a group of practitioners called Defeat Autism Now, which promotes chelation and other complementary/alternative autism treatments, is completing a study of the treatment.
Most of the researchers who spoke with WebMD for this article expressed the opinion that chelation is both ineffective for autism and dangerous; none advise parents to try it.
Gluten-Free Casien-Free (GFCF) Diet for Autism
Many parents of children with autism believe that their children suffer from an inability to digest wheat and/or dairy products. Some who have put their children on gluten-free/casien-free diets report seeing remarkable changes in their children's behavior.
This GFCF diet has become one of the most commonly used treatments for autism, despite concerns that ASD kids -- who tend to be very picky eaters -- may become undernourished by following a GFCF diet.
A highly regarded 1995 study suggested that ASD kids on a GFCF diet for one year had fewer autistic traits. However, preliminary results from a randomized, controlled clinical trial did not show a benefit.
More rigorous randomized, placebo-controlled clinical trials of the GFCF diet -- including one by Hyman -- are under way.
CAM for Autism
Surveys suggest that nine out of 10 parents treat their child's autism with some form of complementary and alternative medicine (CAM). These include both nonbiological treatments such as dolphin-assisted therapy and biological treatments such as dietary supplements.
Most of CAM treatments have either positive parent reports or small, inconclusive studies suggesting they might work. For many, there are inconclusive studies suggesting they are not helpful. In almost all cases, there is no definitive proof that they help, and no rigorous safety studies.
The number of treatments on this list is very large. A list compiled by Hyman includes:
- Dietary restriction of known allergens
- Intravenous immunogloblulins (IVIG)
- Antiviral drugs
- Chelation via DMSA, lipoic acid, clay baths, and natural chelating agents
- Digestive enzymes
- Yeast-free diet
- Antifungal agents
- The Specific Carbohydrate Diet (SCD)
- Antibiotic therapy
- Vitamin B-6 and magnesium
- Vitamin C
- Folic acid
- Vitamin B-12
- Dimethylglycine (DMG)
- Tryptophan and tyrosine supplementation
- Periactin (the antihistamine cyproheptadine)
- Carnosine supplementation
- Omega-3 fatty acids or polyunsaturated fatty acid (PUFA)
- Auditory Integration Training (AIT)
- Behavioral Optometry
- Craniosacral manipulation
- Facilitated communication
In its 2007 guidelines for the management of ASDs, the American Academy of Pediatrics warns that it does not endorse the use of these treatments outside carefully designed, well-monitored clinical trials.
"Unfortunately, families are often exposed to unsubstantiated, pseudoscientific theories and related clinical practices that are, at best, ineffective and, at worst, compete with validated treatments or lead to physical, emotional, or financial harm," the AAP's Council on Children with Disabilities writes.
Progress is being made. Serious researchers are at last responding to parents demands that they evaluate a wide range of autism treatments. And CAM advocacy groups, such as the Defeat Autism Now (DAN) group, are conducting well-respected trials.
One such trial, reported at last years DAN meeting, focused on HBOT -- hyperbaric oxygen therapy -- the latest new CAM autism treatment to emerge. The idea is to put children with autism spectrum disorder into a pressure chamber and push oxygen into their tissues.
"The mechanism of action may not be in keeping with our traditional understanding of brain injury and postnatal treatment in this disorder," Hyman says.
Swedo praises the DAN group for testing this treatment and the study's design. Ultimately, it did not validate HBOT as an autism treatment.
Unfortunately, studies that prove or disprove autism treatments are the exception rather than the rule.
"One of my frustrations is as soon as you think you have a handle on what is worth testing because enough people have used it, another one comes along," Swedo says.
But Hyman warns her fellow researchers about negativity.
"Some things in CAM are very exciting," she says. "Once you demonstrate something works, if doesn't fit into the biological universe you understand, who cares?"
Debbie Page says her experience with her son Gabe brought home to her the importance of starting early with treatments known to be effective -- even if a child's doctors are still arguing about whether the problem is autism or not.
"Just listen to your instinct and you gut," she tells other parents. "No help you get for them is going to hurt them, even if you don't yet have a diagnosis. If your child's communication is not developing, get help for that. You don't need for everyone to agree on a diagnosis to start getting help for your child."